The PNH Registry

The PNH Registry: For All PNH Patients

The PNH Registry is a global, observational, noninterventional study collecting safety, effectiveness, and quality-of-life data on patients with PNH. It was created in response to requests from regulatory authorities at the Food and Drug Administration (FDA) and the European Medicines Agency (EMEA) to collect long-term safety and effectiveness data on patients treated with Soliris®. In its commitment to patients, Alexion extended the charge of the registry to collect safety and additional information on patients, whether treated with Soliris or not, in order to enhance the understanding of the disease.

PNH Registry objectives: optimize patient management and outcomes

  • To enhance the understanding of PNH demographics and natural history
  • To capture the long-term outcomes of patients in order to better guide and assess therapeutic interventions and the safety of Soliris
  • To serve as a global resource for PNH information

The PNH Registry is designed to protect the confidentiality of patient data in accordance with all applicable privacy regulations. All patients diagnosed with PNH can participate.

Benefits of participation: helping physicians and patients

The PNH Registry brings together physicians and patients from around the world. You will be joining a global community of physicians contributing to the largest, most comprehensive database on PNH.

Additional benefits of the PNH Registry include:

  • Enhancing the understanding of PNH diagnosis and treatment
  • Helping define treatment objectives, practice patterns, and best practices
  • Promoting evidence-based medicine
  • Promoting scientific collaboration in the PNH community
  • Providing an opportunity to participate in periodic registry meetings to share data and treatment experience

Because PNH is a rare disorder, the experience of every patient counts toward advancing and understanding the disease while helping to raise awareness of PNH in the medical community at large—please consider participating.

Who can contribute to the PNH Registry?

  • All physicians treating PNH patients—regardless of therapy
  • All patients who have been diagnosed with PNH are eligible to be enrolled
  • All patients receiving Soliris are eligible to be enrolled

Data are entered at enrollment and approximately every 6 months thereafter. Physicians are not required to examine patients at that time. Data entry minimally includes: demographics, medical history, PNH diagnosis, flow cytometry, symptomology, safety events, clinical outcomes, and pregnancy. Physicians will receive compensation for participation.

The PNH Registry is sponsored by Alexion Pharmaceuticals and managed by ICON Clinical Research.

Please contact the PNH Registry hotline:

Email: PNHregistry@iconplc.com

Telephone: 1.800.913.4893

International: +1 215.616.3558





IMPORTANT SAFETY INFORMATION

WARNING: SERIOUS MENINGOCOCCAL INFECTIONS

Soliris increases the risk of meningococcal infections. Meningococcal infection may become rapidly life-threatening or fatal if not recognized and treated early

  • Vaccinate patients with a meningococcal vaccine at least 2 weeks prior to receiving the first dose of Soliris; revaccinate according to current medical guidelines for vaccine use
  • Monitor patients for early signs of meningococcal infections, evaluate immediately if infection is suspected, and treat with antibiotics if necessary

The effect of withdrawal of anticoagulant therapy during Soliris treatment has not been established. Therefore, treatment with Soliris should not alter anticoagulant management.

The most frequent adverse events observed in clinical studies were headache, nasopharyngitis, back pain, nausea, and fatigue.

SEE FULL PRESCRIBING INFORMATION FOR COMPLETE BOXED WARNING INCLUDING WARNINGS, PRECAUTIONS, AND ADVERSE REACTIONS.